NCT05906953招募中1 期
A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects With RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)
HuidaGene Therapeutics Co., Ltd.1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2023年10月31日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Incidence of ocular and non-ocular adverse events
研究概览
简要总结
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or females between 6 and 50 years of age at the time of signing the informed consent form.
- •Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.
- •Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.
- •Ability to perform tests of visual and retinal function.
- •Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
- •Acceptable hematology, clinical chemistry, and urine laboratory parameters.
排除标准
- •Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).
- •Presence of epiretinal membrane by OCT.
- •Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
- •Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
- •Prior ocular surgery within six months.
- •Prior gene therapy or oligonucleotide therapy treatments.
- •Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.
研究组 & 干预措施
HG004
Experimental
干预措施: HG004 (Drug)
结局指标
主要结局
Incidence of ocular and non-ocular adverse events
时间窗: 52 weeks
次要结局
- Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.(52 weeks)
- Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart(52 weeks)
研究者
研究点 (1)
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